Ocugen (OCGN) Chardan's 8th Annual Genetic Medicines Conference summary

Company overview and technology platform

• Focuses on therapies for unmet medical needs in retinal diseases, orthopedic injuries, and respiratory illnesses.

• Utilizes a modified gene therapy platform targeting multiple mutations and disease pathophysiology, not just single-gene defects.

• Platform is novel in ophthalmology, offering a disease-modifying approach rather than traditional gene augmentation or editing.

OCU400 for retinitis pigmentosa (RP) and LCA

• OCU400 targets a broad RP population by addressing cellular and molecular imbalances, not just specific mutations.

• Only one approved RP therapy exists, covering 1-2% of patients; OCU400 aims for broader applicability.

• Phase I/II trials showed good safety and efficacy across multiple mutations, with durable benefits up to 12 months.

• No clear dose response observed, likely due to the transcription factor mechanism.

• Phase III liMeliGhT trial is enrolling 150 patients, split between RHO and gene-agnostic arms, with a primary endpoint of LDNA; BLA planned for 2026.

OCU410 and OCU410ST for geographic atrophy and Stargardt disease

• OCU410 targets multiple pathways in geographic atrophy, including complement, lipid peroxidation, inflammation, and oxidative stress.

• RORA gene therapy demonstrated reduction in drusen and anti-inflammatory effects in preclinical models.

• Phase I/II trial for OCU410 completed phase I dosing; phase II is ongoing with 45 subjects, and initial data expected by year-end.

• OCU410ST for Stargardt disease completed phase I; awaiting six-month data to inform phase II design.